Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs

• DOI: 10.1016/j.omtn.2022.12.006
• Publication Date: 2022-12-09T16:49:43Z
• Authors: Ortal Iancu; Daniel Allen; Orli Knop; Yonathan Zehavi; Dor Breier; Adaya Arbiv; Atar Lev; Yu Nee Lee; Katia Beider; Arnon Nagler; Raz Somech; Ayal Hendel
• Keywords: Multiplex

Severe combined immunodeficiency (SCID) is a group of disorders caused by mutations in genes involved the process lymphocyte maturation and function. CRISPR-Cas9 gene editing patient's own hematopoietic stem progenitor cells (HSPCs)